There are a number of basic standards of diagnosis and care that need to be implemented across Europe.
- We must ensure that CF is diagnosed early throughout Europe, preferably by newborn screening; and that everyone in Europe has access to appropriate diagnostic testing, performed by experienced personnel.
- We must ensure that every CF patient is genotyped, in order to determine eligibility for novel small molecule therapies.
- Having been diagnosed, it is essential that from diagnosis, every CF patient in Europe has access to CF specialist centre care by the full MDT and all necessary medications. This last is an ambitious goal because CF medications are expensive and currently many European countries cannot afford them.
- On reaching adulthood, it is essential that patients are treated in a CF unit experienced in CF care. This is a pressing need: adult CF clinic development has lagged behind paediatrics in many parts of Europe.
It is clear that the predictions in the previous edition of this book have not all been fulfilled! We should aim to achieve the following:
- Universal newborn screening in Europe.
- Evidence-based care for newborn-screened babies, driven by the performance of randomised controlled trials with appropriate end- points. There is currently only one (a negative trial of hypertonic saline) that is of a satisfactory standard. This aim is in the mainstream of the European Union drive for medicines for children.
- Access to all evidence-based CF medications across Europe, eliminating regional inequalities.
- All European CF patients being seen regularly at a fully staffed, fully equipped CF centre; this is particularly necessary, and particularly far from being achieved, in adult patients. There is clear evidence that specialist centre care is beneficial.
- Increased use of care in the community and telemedicine to minimise hospital contacts.
- A fully funded, comprehensive registry which captures data on all European patients.
Recent trends will necessitate changes in the provision of care:
- Increased longevity will mean a radical rethink about the burden of care. A well adult who happens to have CF with minimal impact is not likely to want to take many therapies or perform as much airway clearance as the sick CF patient.
- Increased longevity will also mean we need to detect and deal with new emerging infections and new iatrogenic complications, such as antibiotic allergy and subtle long-term medication side-effects.
- New treatments will increase the financial costs of care: new products will build on old concepts, such as novel inhaled antibiotics and mucolytics; more therapies will address the basic defect (gene therapy, for example) and PTC124 for class I mutations.
- More attention must be paid to extrapulmonary complications. There will be pressure on diabetic and endocrine clinics, and obstetric services for pregnant women with CF.
- There will be increased demands for lung transplant services, which will mean optimising the donor supply, novel techniques to salvage donor lungs previously thought to be untransplantable, living related donation, and ultimately a transgenic large mammalian source.